Objective: Evaluate the feasibility and reliability of remote assessments of adult individuals with CMT1A to enable remote monitoring of participants in clinical trials.
Background: Recent knowledge gains regarding the genetics, pathomechanisms and natural history of CMT1A, as well as emerging therapeutic candidates, highlight the need to prepare for clinical trials by identifying valid and responsive outcome measures. Grip dynamometry and the 9-hole peg test (9HPT) measure upper limb function and the timed up and go (TUG) assesses gait and balance function – deficits in these areas commonly cause disability in CMT1A. Remote administration of these assessments, if found reliable, can reduce participant burden, increase participation of those living further from research centers, and provide frequent monitoring during clinical trials.
Methods: After an in-person visit, participants were sent home with a Jamar Plus digital hand dynamometer, 9HPT, and 3-meter tape for the TUG. Within one week of the in-person visit, a remote assessment was performed by the clinical evaluator via synchronous HIPPA compliant video conferencing. Time to complete the 9HPT and TUG was recorded, as well as 3 trials of grip dynamometry. Feasibility was assessed and results of the in-person and remote assessments were compared.
Results: Fifteen participants (ages 18-64) completed all of the remote assessments, which took 5-10 minutes. Both the TUG and 9HPT demonstrated a 0.91 ICC (0.75-0.97 CI; p <0.0001). Hand grip dynamometry had an ICC of 0.87 (0.64-0.96 CI; p <0.0001).
Conclusion: Remote assessments took minimal time to complete and were feasible in this pilot study. The TUG and 9HPT demonstrate excellent reliability and grip dynamometry shows good reliability. Reliability of the handgrip may have been limited by the use of two different dynamometers. Examining the feasibility and reliability of remote assessments in a larger population and over time is necessary to determine the utility in clinical trials.